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The clinical trial dilemma

The clinical trial dilemma
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First Published: Mon, Feb 12 2007. 11 59 PM IST
Updated: Mon, Feb 12 2007. 11 59 PM IST
On 1 March, a US federal appeals court will hear oral arguments in the case of the Abigail Alliance organization’s lawsuit to change systems at the Food and Drug Administration (FDA) to allow terminally ill patients access to promising drugs that have successfully completed initial stages of human safety testing. Because of my former role in the oncology division at FDA, and in my eight-year experience as a cancer patient advocate on behalf of my son, I may be able to shed some light on the regulatory policy, medical drug development and patient rights issues surrounding this landmark case.
Abigail Burroughs was a young lady who developed a rare form of cancer in her mouth. As she was dying, her father fought to gain access to a drug that could possibly help her, but was denied access because the drug had not yet been approved by FDA. After Abigail died, her father formed the Abigail Alliance, an organization which subsequently sued the government to force change in the policies that require FDA control over which dying patient does and does not receive approval to receive a drug not yet on the market. In the first round in the courts, FDA won but, on appeal, the Abigail Alliance won. 1 March is the rubber match of the lawsuit.
If the Abigail Alliance is successful, drug companies could seek early approval of a drug for patients who are terminally ill and who are ineligible for participation in human testing of drugs that occur prior to FDA approval. The only requirement would be that the drug must have surpassed the initial safety phase of human testing, called “Phase I clinical trials.” Critics of that position claim that current regulations and processes fully meet the needs of such patients—doctors merely need to ask the government, on behalf of their patients, for access to any unapproved drug they wish.
If these critics could be a fly on the wall at FDA after such a special request is made by a doctor, they might change their tune. I can attest to the burden the physician-sponsors of these requests have to go through with FDA reviewers as they run the gauntlet. Manufacturing, pharmacology, toxicology, pharmacokinetic, clinical and even statistical “issues” raised by FDA staff, aimed at the applying physician, can sometimes rival receipt of an audit from the Internal Revenue Service (IRS). Requests are on occasion withdrawn by exasperated doctors or refused by the FDA, leaving patients to fend for themselves.
Other critics of Abigail Alliance also claim that at the threshold for early approval for the terminally ill, i.e., post-Phase I testing, the safety of the drug is still very much a concern; many more years of snuggling with the data will still be necessary before FDA will feel things are just right. The fact is that all drugs, no matter what stage of development, have the potential to evolve new safety concerns. Since a drug is safe enough for the hundreds of patients in the “Phase II” of human testing, how is it not safe enough for a patient whose only option is the terminal progression of his disease?
Critics of early access also present doomsday scenarios of the impact of success of the Abigail Alliance case on the medical drug development process called “Phase III clinical trials.” Over the last 25 years, the US has evolved an enormous engine for testing new drugs on patients at hundreds of medical centres in the country. If you have a life-threatening disease like cancer, and you want to try a drug not yet in the market, you better belly-up to a clinical trial, or wait a few years before your doctor can prescribe it. It is a bloated and slow engine, but it is the best process we have to try to show if a new drug works or not.
Some with a vested interest in maintaining the status quo, such as medical society groups, might feel threatened by a victory of the Abigail Alliance if the perception is that a patient will be able to easily opt out of clinical trials and just get the drug directly. How will we ever get new drugs properly tested if everyone is slipping past the engine? In my opinion, this threat is non-existent. One criterion of the patients under consideration by the court is that they cannot even be eligible for a clinical trial. In addition, the FDA will still have enormous authority in deciding who is truly terminal, who is eligible for other clinical trials, and whether other treatment options exist.
And what about the rights of the dying patient in all this? In the case of those with cancer, the patient turns not to FDA but to his oncologist for options. These physicians are among the most well-educated and well-intentioned individuals in our society. The oncologist already has much access to off-label use of approved medications for patients in a terminal setting. The system is well suited to adapting, should an Abigail Alliance victory add additional options for the patients and physicians faced with advancing and otherwise untreatable cancer.
Patients have valid arguments in demanding greater access to promising agents under development. Public servants should respect citizens who advocate that they be allowed to have a say in methods of their treatment when terminally ill, and government officials should have very compelling reasons for denying such access. New drug development will not suffer if a small minority of patients fighting for their lives, with no other options and in concert with their physician, gain access to a potentially beneficial agent with an established basic safety profile.
Mark Thornton was formerly a medical reviewer in the oncology division at the FDA and is currently a senior vice-president for GenVec, Inc. The Wall Street Journal.
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First Published: Mon, Feb 12 2007. 11 59 PM IST
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